Betibeglogene Autotemcel Gene Therapy for Non-β0/β0 Genotype β-Thalassemia.

doi:10.1056/NEJMoa2113206

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Betibeglogene Autotemcel Gene Therapy for Non-β0/β0 Genotype β-Thalassemia.

The New England journal of medicine - 3 Feb 2022

Locatelli Franco, Thompson Alexis A, Kwiatkowski Janet L, Porter John B, Thrasher Adrian J, Hongeng Suradej, Sauer Martin G, Thuret Isabelle, Lal Ashutosh, Algeri Mattia, Schneiderman Jennifer, Olson Timothy S, Carpenter Ben, Amrolia Persis J, Anurathapan Usanarat, Schambach Axel, Chabannon Christian, Schmidt Manfred, Labik Ivan, Elliot Heidi, Guo Ruiting, Asmal Mohammed, Colvin Richard A, Walters Mark C

Abstract excerpt

BACKGROUND: Betibeglogene autotemcel (beti-cel) gene therapy for transfusion-dependent β-thalassemia contains autologous CD34+ hematopoietic stem cells and progenitor cells transduced with the BB305 lentiviral vector encoding the β-globin (βA-T87Q) gene. METHODS: In this open-label, phase 3 study, we evaluated the efficacy and safety of beti-cel in adult and pediatric patients with transfusion-dependent...

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PMID: 34891223
DOI: 10.1056/NEJMoa2113206

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